FDA Expediting Review of Regeneron Drug to Treat Genetic Hearing Loss
Regeneron’s DB-OTO gene therapy for genetic hearing loss is one of nine drugs to receive expedited review under FDA's new CNPV program.
Tag: otoferlin-deficiency
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Akouos’ AK-OTOF Receives Orphan Drug Designation
Akouos, Inc announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on the company’s application for orphan drug designation for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. The positive opinion was subsequently adopted by the European Commission.
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Sensorion and Pasteur Institute to Collaborate on Gene Therapy Products
Sensorion announced a letter of intent with Pasteur Institute in Paris to exclusively negotiate a framework agreement to obtain the exclusive licenses to develop and commercialize gene therapy product candidates for restoration, treatment, and prevention of hearing loss disorders. They will collaborate on several lead programs to correct monogenic forms of hereditary hearing loss including, among others, the Usher syndrome type 1 and otoferlin deficiency, according to the announcement.